Proton pump inhibition for secondary hemochromatosis in hereditary anemia, a phase III placebo-controlled randomized cross-over clinical trial. PPI Shine Again Maagzuurremmers voor de behandeling van secundaire hemochromatose bij patiënten met erfelijke niet-transfusie afhankelijke bloedarmoedes.

DOI

Iron overload is a severe general complication of hereditary anemias. Treatment with iron chelators is hampered by important side-effects, high costs and the lack of availability in many countries with high prevalence of hereditary anemias. Objective: to investigate the safety and efficacy of esomeprazole in treatment of iron overload in patients with non-transfusion-dependent hereditary anemias. Design: phase III randomized placebo-controlled cross-over trial. Setting: enrollment from March 2018 through April 2019 in five centers of expertise in the Netherlands. Participants: 30 adult patients with mild-to-moderate iron overload were enrolled (non-transfusion-dependent thalassemia (N=13), pyruvate kinase deficiency (N=8), congenital sideroblastic anemia (N=3), congenital dyserythropoietic anemia (N=3), sickle cell disease (N=2), and hereditary elliptocytosis (N=1)). Intervention: esomeprazole 40mg BID and placebo for 12 months in a cross-over design. Primary outcome: change of liver iron content (LIC) measured by MRI.

Metadata are included in the repository. Please send a request to the Data Access Committee for access to the full data set.

Identifier
DOI https://doi.org/10.34894/PT05Q8
Metadata Access https://dataverse.nl/oai?verb=GetRecord&metadataPrefix=oai_datacite&identifier=doi:10.34894/PT05Q8
Provenance
Creator van Vuren, Annelies ORCID logo
Publisher DataverseNL
Contributor van Vuren, Annelies; van Beers, Eduard; Vianen, Marieke; UMC Utrecht
Publication Year 2021
Funding Reference ZonMw; Innovatiefonds Zorgverzekeraars
Rights The standard Data Sharing Agreement (DSA) of the UMC Utrecht must be signed without adjustments. This DSA is in compliance with Dutch law. No costs are involved.; info:eu-repo/semantics/openAccess
OpenAccess true
Contact van Vuren, Annelies (umcutrecht.nl); van Beers, Eduard (umcutrecht.nl); Vianen, Marieke (umcutrecht.nl)
Representation
Resource Type Dataset
Format text/csv; application/pdf
Size 5544; 358425; 5058; 59136; 23906
Version 1.3
Discipline Life Sciences; Medicine